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En fase 3-studie av fenfluraminhydroklorid ved Rett-syndrom

30. oktober 2026 oppdatert av: UCB BIOSCIENCES, Inc.

En fase 3 randomisert, dobbeltblind, placebokontrollert, parallell gruppe, multicenter studie med åpen etikett-utvidelse for å evaluere effektiviteten og sikkerheten til Fenfluramin Hydroklorid i studie-deltakere med Rett syndrom

The purpose of this study is to investigate the efficacy of fenfluramine hydrochloride (HCl) versus placebo in study participants with Rett syndrome (RTT).

Studieoversikt

Status

Rekruttering

Forhold

Intervensjon / Behandling

Studietype

Intervensjonell

Registrering (Antatt)

200

Fase

  • Fase 3

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiekontakt

Studer Kontakt Backup

Studiesteder

  • Belgia
      • Brussels, Belgia
        • Rekruttering
        • Ep0247 11001
      • Edegem, Belgia
        • Rekruttering
        • Ep0247 11002
  • Forente stater
    • Arkansas
      • Little Rock, Arkansas, Forente stater, 72202
        • Rekruttering
        • Ep0247 21010
    • Florida
      • Miami, Florida, Forente stater, 33155
        • Rekruttering
        • Ep0247 21009
      • Orlando, Florida, Forente stater, 32806
        • Rekruttering
        • Ep0247 21011
    • Georgia
      • Atlanta, Georgia, Forente stater, 30322
        • Rekruttering
        • Ep0247 21007
    • Pennsylvania
      • Philadelphia, Pennsylvania, Forente stater, 19104-4318
        • Rekruttering
        • Ep0247 21003
    • Texas
      • Houston, Texas, Forente stater, 77030
        • Rekruttering
        • Ep0247 21005
      • Irving, Texas, Forente stater, 75062
        • Rekruttering
        • Ep0247 21013
    • Washington
      • Seattle, Washington, Forente stater, 98105
        • Rekruttering
        • Ep0247 21004
  • Frankrike
      • Brest, Frankrike
        • Rekruttering
        • Ep0247 12005
      • Bron, Frankrike
        • Rekruttering
        • Ep0247 12003
      • Marseille, Frankrike
        • Rekruttering
        • Ep0247 12004
      • Paris, Frankrike
        • Rekruttering
        • Ep0247 12006
      • Paris, Frankrike
        • Rekruttering
        • Ep0247 12001
  • Georgia
      • Tbilisi, Georgia
        • Rekruttering
        • Ep0247 13001
      • Tbilisi, Georgia
        • Rekruttering
        • Ep0247 13002
  • Israel
      • Ramat Gan, Israel
        • Rekruttering
        • Ep0247 16001
  • Italia
      • Genova, Italia
        • Rekruttering
        • Ep0247 17003
      • Milan, Italia
        • Rekruttering
        • Ep0247 17001
      • Naples, Italia
        • Rekruttering
        • Ep0247 17008
      • Pisa, Italia
        • Rekruttering
        • Ep0247 17002
      • Troina, Italia
        • Rekruttering
        • Ep0247 17006
  • Japan
      • Chiyoda-ku, Japan
        • Rekruttering
        • Ep0247 43005
      • Kodaira, Japan
        • Rekruttering
        • Ep0247 43004
      • Kurume, Japan
        • Rekruttering
        • Ep0247 43001
      • Musashimurayama-shi, Japan
        • Rekruttering
        • Ep0247 43006
      • Osaka, Japan
        • Rekruttering
        • Ep0247 43002
      • Setagaya-ku, Japan
        • Rekruttering
        • Ep0247 43003
  • Polen
      • Gdansk, Polen
        • Rekruttering
        • Ep0247 19003
      • Krakow, Polen
        • Rekruttering
        • Ep0247 19002
  • Spania
      • Madrid, Spania
        • Rekruttering
        • Ep0247 11105
      • Madrid, Spania
        • Rekruttering
        • Ep0247 11101
      • Málaga, Spania
        • Rekruttering
        • Ep0247 11103
      • Seville, Spania
        • Rekruttering
        • Ep0247 11102
  • Storbritannia
      • Manchester, Storbritannia
        • Rekruttering
        • Ep0247 11303
      • Nottingham, Storbritannia
        • Rekruttering
        • Ep0247 11304
  • Tyskland
      • Vogtareuth, Tyskland
        • Rekruttering
        • Ep0247 14001
  • Ungarn
      • Budapest, Ungarn
        • Rekruttering
        • Ep0247 15001
      • Debrecen, Ungarn
        • Rekruttering
        • Ep0247 15002

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

5 år til 35 år (Barn, Voksen)

Tar imot friske frivillige

Nei

Kjønn som er kvalifisert for studier

Alle

Beskrivelse

Inclusion Criteria:

  • Participant has typical or classic Rett Syndrome (RTT) according to the RettSearch Consortium 2010 revised criteria
  • Participant has a documented disease-causing mutation or deletion in the methyl-CpG-binding protein 2 (MECP2) gene
  • Participant meets criteria for postregression for at least 6 months prior to Screening, defined as:
  • No loss or degradation of ambulation (including gait, coordination, or independence of walking/standing);
  • No loss or degradation of hand function; no loss or degradation of speech (including babbling, words, or previously developed communicative vocalizations);
  • No loss or degradation of nonverbal communicative or social skills (including eye gaze, using body to indicate communicative intent, or social attentiveness)
  • Participant has an Rett Syndrome Clinical Severity Scale (RTT-CSS) rating of 10 to 36 (inclusive)
  • Participant has a Clinical Global Impression-Severity (CGIS) score of ≥4
  • Participant has a legal representative capable of providing signed informed consent on behalf of the participant as described in the protocol, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol.
  • Participant is aged 5 to 35 years of age (inclusive) at the time of first administration of investigational intervention.
  • Male or female.
  • Participant has a consistent caregiver who is ≥18 years of age at the Screening Visit. The caregiver needs to be able to complete the caregiver assessments defined for the entire study. Every attempt should be made to have the same evaluator complete the assessments for the duration of the study.

Exclusion Criteria:

  • Participant has a history of lymphoma, leukemia, or any malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for 3 years.

  • Participant has clinically significant abnormality in vital signs according to the Investigator

  • Participant has an exclusionary cardiovascular or cardiopulmonary abnormality based on echocardiogram (ECHO), electrocardiogram (ECG), or physical examination, and is not approved for entry by the central cardiac reader. Exclusionary abnormalities include, but are not limited to:

    1. Greater than trace aortic valve regurgitation.
    2. Greater than mild mitral valve regurgitation.
    3. Possible signs of pulmonary arterial hypertension (PAH) with abnormal pulmonary artery systolic pressure (PASP) or PASP ≥35 mmHg.
    4. Evidence of left ventricular dysfunction (systolic or diastolic).
    5. Clinically significant structural cardiac abnormality, including but not limited to mitral valve prolapse, atrial or ventricular septal defects, or patent ductus arteriosus with reversal of shunt (right to left shunt). Note: Patent foramen ovale without a reversal of shunt or a bicuspid aortic valve is not considered exclusionary

  • Participant has a clinically significant medical condition, including chronic obstructive pulmonary disease, interstitial lung disease, portal hypertension, or need for invasive mechanical ventilation (eg, via tracheostomy), or has had clinically relevant symptoms or a clinically significant illness currently or in the 4 weeks prior to the Screening Visit that would negatively impact study participation, collection of study data, or pose a risk to the participant

  • Participant is taking >4 concomitant antiseizure medications (ASMs). Rescue medications are not included in the count

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: Randomisert
  • Intervensjonsmodell: Parallell tildeling
  • Masking: Trippel

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: fenfluramine hydrochloride

Participants will receive fenfluramine hydrochloride for 14 weeks in a double-blind period (including titration and maintenance), followed by a 2-week double-blind transition period. This is followed by a 52-week open-label treatment period. Participants may continue treatment until alternative access is available. Those who discontinue without continued access will undergo an 8-day taper and a 26-week follow-up.
Legemiddel: fenfluramine hydrochloride

Oral solution

Andre navn:
  • Fintepla, ZX008
Placebo komparator: Placebo

Participants will receive a matching placebo for 14 weeks in a double-blind period (including titration and maintenance). After this period, they transition into a 2-week double-blind period with fenfluramine hydrochloride, followed by a 52-week open-label treatment period. Participants may continue treatment until alternative access is available. Those who discontinue without continued access will undergo an 8-day taper and a 26-week follow-up.
Annen: Placebo

Oral solution

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Change from Baseline to Week 14 in Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score
Tidsramme: From Baseline (Day 1) to Week 14

The RSBQ is a caregiver-completed, instrument assessing behavioral and emotional features in RTT. The RSBQ consists of 45 items, including 8 subscales: General mood (8 items); Breathing problems (5 items); Hand behavior (6 items); Face movements (4 items); Body rocking and expressionless face (6 items); Nighttime behaviors (3 items); Fear/anxiety (4 items); and Walking/standing (2 items). Caregivers are asked to evaluate each RTT feature based on the current status of the patients on a 3-point scale as 0 ("not true"), 1 ("somewhat or sometimes true"), or 2 ("often true"), with the total score ranging from 0 to 90. Higher scores indicate increased disease severity. Seven items that do not belong under any of the subscales are classed as "uncategorized" but contribute to the overall total score.
From Baseline (Day 1) to Week 14
Clinical Global Impression of Change (CGIC) Score at Week 14
Tidsramme: At Week 14

The CGIC is a clinician-rated single item evaluating the degree of improvement or worsening of a participant's condition from Baseline following treatment or intervention. The CGIC uses a 7-point response scale, with the following ratings: "1: Very Much improved", "2: Much improved", "3: Minimally improved", "4: No change", "5: Minimally worse", "6: Much worse", "7: Very Much Worse".
At Week 14

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Change from Baseline to Week 14 in Patient-Reported Outcomes Measurement Information System-Sleep Disturbance (PROMIS-SD) score
Tidsramme: From Baseline (Day 1) to Week 14

Sleep disturbances will be assessed by the PROMIS-SD parent proxy 8a version. Caregivers are asked to rate 8 items over the past 7 days on a 5-point scale: "1: Never", "2: Almost never", "3: Sometimes", "4: Almost always", and "5: Always", with higher scores indicating more severe sleep disturbances.
From Baseline (Day 1) to Week 14
Change from Baseline to Week 14 in Observer-Reported Communication Ability (ORCA) score
Tidsramme: From Baseline (Day 1) to Week 14

The ORCA is an 84-item, observer-reported measure of communication ability over the past 30 days. The majority of the items included in the ORCA measure have 3 response options: "No or only once," "Sometimes," and "Yes, almost all the time", which enable derivation of an overall communication score and scores for each form of communication (expressive, receptive, and pragmatic), with higher scores indicating greater communication ability.
From Baseline (Day 1) to Week 14
Caregiver Global Impression of Change - Seizure (CaGIC-Seizure) score at Week 14
Tidsramme: At Week 14

The CaGIC-Seizure is a caregiver-reported item assessing the change from Baseline in the participant's seizure status. The CaGIC-Seizure uses a 7-point response scale, with the following ratings: "1: Very Much improved", "2: Much improved", "3: Minimally improved", "4: No change", "5: Minimally worse", "6: Much worse", "7: Very Much Worse".
At Week 14
Incidence of treatment-emergent adverse events (TEAEs) during the double-blind intervention period
Tidsramme: From Baseline (Day 1) up to Week 14

An AE is any untoward medical occurrence in a clinical study participant temporally associated with the use of study intervention, whether or not considered related to the study intervention. Treatment emergent adverse events (TEAEs) are adverse events that are not present prior to the pharmaceutical product administration or an already present event that worsens either in intensity or frequency.
From Baseline (Day 1) up to Week 14
Incidence of serious TEAEs during the double-blind intervention period
Tidsramme: From Baseline (Day 1) up to Week 14

An SAE is defined as any untoward medical occurrence that, at any dose, meets 1 or more of the criteria listed:

  • Results in death
  • Is life-threatening
  • Requires inpatient hospitalization or prolongation of existing hospitalization
  • Results in persistent or significant disability/incapacity
  • Is a congenital anomaly/birth defect
  • Other important medical events which based on medical or scientific judgement may jeopardize the patients or may require medical or surgical intervention to prevent any of the above.
From Baseline (Day 1) up to Week 14
Incidence of TEAEs leading to discontinuation during the double-blind intervention period
Tidsramme: From Baseline (Day 1) up to Week 14

An AE is any untoward medical occurrence in a clinical study participant temporally associated with the use of study intervention, whether or not considered related to the study intervention. Treatment emergent adverse events (TEAEs) are adverse events that are not present prior to the pharmaceutical product administration or an already present event that worsens either in intensity or frequency. TEAEs leading to discontinuation will be reported.
From Baseline (Day 1) up to Week 14
Incidence of related TEAEs during the double-blind intervention period
Tidsramme: From Baseline (Day 1) up to Week 14

An AE is any untoward medical occurrence in a clinical study participant temporally associated with the use of study intervention, whether or not considered related to the study intervention. Treatment emergent adverse events (TEAEs) are adverse events that are not present prior to the pharmaceutical product administration or an already present event that worsens either in intensity or frequency. Related TEAEs will be reported.
From Baseline (Day 1) up to Week 14
Change from Baseline in QT interval corrected using Fridericia's formula (QTcF) interval on 12-lead ECG by visit during the double-blind intervention period
Tidsramme: From Baseline (Day 1) up to Week 14

Change from Baseline in QTcF interval as measured by 12-lead ECG up to Week 14 by visit will be reported
From Baseline (Day 1) up to Week 14
Incidence of treatment-emergent Doppler Echocardiogram (ECHO) results meeting the Food and Drug Administration (FDA) case definition of drug-associated valvular heart disease (VHD) during the double-blind intervention period
Tidsramme: From Baseline (Day 1) up to Week 14

The FDA case definition of drug-associated VHD is aortic regurgitation ≥mild and/or mitral regurgitation ≥moderate with restricted valve motion, valve thickening, and/or physical signs or symptoms attributable to valve diseases. ECHO readings related to VHD on any of the 4 valves (aortic, mitral, pulmonary, tricuspid) will be reported with grades of absent, trace, mild, moderate, or severe.
From Baseline (Day 1) up to Week 14
Incidence of treatment-emergent Doppler ECHO results meeting the FDA case definition of PAH (defined as a PASP >35mmHg) during the double-blind intervention period
Tidsramme: From Baseline (Day 1) up to Week 14

ECHO readings related to pulmonary arterial hypertenstion (PAH) will be reported based on Pulmonary artery systolic pressure (PASP).
From Baseline (Day 1) up to Week 14
Incidence of treatment-emergent adverse events (TEAEs) from baseline to end of safety follow up
Tidsramme: From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months)

An AE is any untoward medical occurrence in a clinical study participant temporally associated with the use of study intervention, whether or not considered related to the study intervention. Treatment emergent adverse events (TEAEs) are adverse events that are not present prior to the pharmaceutical product administration or an already present event that worsens either in intensity or frequency.
From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months)
Incidence of serious TEAEs from baseline to end of safety follow up
Tidsramme: From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months)

An SAE is defined as any untoward medical occurrence that, at any dose, meets 1 or more of the criteria listed:

  • Results in death
  • Is life-threatening
  • Requires inpatient hospitalization or prolongation of existing hospitalization
  • Results in persistent or significant disability/incapacity
  • Is a congenital anomaly/birth defect
  • Other important medical events which based on medical or scientific judgement may jeopardize the patients or may require medical or surgical intervention to prevent any of the above.
From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months)
Incidence of TEAEs leading to discontinuation from baseline to end of safety follow up
Tidsramme: From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months)

An AE is any untoward medical occurrence in a clinical study participant temporally associated with the use of study intervention, whether or not considered related to the study intervention. Treatment emergent adverse events (TEAEs) are adverse events that are not present prior to the pharmaceutical product administration or an already present event that worsens either in intensity or frequency. TEAEs leading to discontinuation will be reported.
From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months)
Incidence of related TEAEs from baseline to end of safety follow up
Tidsramme: From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months)

An AE is any untoward medical occurrence in a clinical study participant temporally associated with the use of study intervention, whether or not considered related to the study intervention. Treatment emergent adverse events (TEAEs) are adverse events that are not present prior to the pharmaceutical product administration or an already present event that worsens either in intensity or frequency. Related TEAEs will be reported.
From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months)
Change from Baseline in QT interval corrected using Fridericia's formula (QTcF) interval on 12-lead ECG by visit from baseline to end of safety follow up
Tidsramme: From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months)

Change from Baseline in QTcF interval as measured by 12-lead ECG up to Week 98 by visit will be reported
From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months)
Incidence of treatment-emergent Doppler Echocardiogram (ECHO) results meeting the Food and Drug Administration (FDA) case definition of drug-associated valvular heart disease (VHD) from baseline to end of safety follow up
Tidsramme: From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months)

The FDA case definition of drug-associated VHD is aortic regurgitation ≥mild and/or mitral regurgitation ≥moderate with restricted valve motion, valve thickening, and/or physical signs or symptoms attributable to valve diseases. ECHO readings related to VHD on any of the 4 valves (aortic, mitral, pulmonary, tricuspid) will be reported with grades of absent, trace, mild, moderate, or severe.
From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months)
Incidence of treatment-emergent Doppler ECHO results meeting the FDA case definition of PAH (defined as a PASP >35mmHg) from baseline to end of safety follow up
Tidsramme: From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months)

ECHO readings related to pulmonary arterial hypertenstion (PAH) will be reported based on Pulmonary artery systolic pressure (PASP).
From Baseline (Day1) to the End of Safety Follow Up (up to 3 years and 10 months)

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

UCB BIOSCIENCES, Inc.

Etterforskere

  • Studieleder: UCB Cares, 0018445992273

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Faktiske)

14. juli 2026

Primær fullføring (Antatt)

6. februar 2029

Studiet fullført (Antatt)

29. august 2031

Datoer for studieregistrering

Først innsendt

25. mars 2026

Først innsendt som oppfylte QC-kriteriene

25. mars 2026

Først lagt ut (Faktiske)

31. mars 2026

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

2. november 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

30. oktober 2026

Sist bekreftet

1. oktober 2026

Mer informasjon

Begreper knyttet til denne studien

Nøkkelord

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • EP0247
  • 2025-523157-34 (Annen identifikator: EU CTR)
  • 2025 (U.S. NIH-stipend/kontrakt)

Plan for individuelle deltakerdata (IPD)

Planlegger du å dele individuelle deltakerdata (IPD)?

JA

IPD-planbeskrivelse

Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.

IPD-delingstidsramme

Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe or global development is discontinued, and 18 months after trial completion.

Tilgangskriterier for IPD-deling

Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal.

IPD-deling Støtteinformasjonstype

  • STUDY_PROTOCOL
  • SEVJE
  • CSR

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Ja

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .

Abonnere